Phase 2: In Vivo Efficacy Studies

Phase 2: In Vivo Efficacy Studies

Details:

Animal Models:

  • Theranib expanded preclinical evaluations to mouse models of breast cancer, focusing on tumors with high ALDH1A3 expression.
  • Models were designed to closely mimic human tumor microenvironments, ensuring clinically relevant findings.
  • Studies included controls and varying VE3 doses to assess its efficacy and safety across a range of conditions.

Tumor Growth Suppression:

  • VE3 demonstrated a statistically significant reduction in tumor volume in treated mice compared to controls.
  • The compound effectively targeted ALDH1A3-positive tumor cells, confirming its ability to inhibit cancer progression.
  • Additional analyses revealed reduced metastatic spread to secondary organs, highlighting VE3’s potential to address metastasis.

Tolerability Studies:

  • Toxicology assessments confirmed that VE3 was well-tolerated at therapeutic doses, with no adverse effects observed in animal behavior, weight, or organ function.
  • Histological evaluations of major organs (e.g., liver, kidney) showed no significant toxicity, reinforcing the compound's safety profile.
  • Repeat-dose studies indicated that VE3 could be safely administered over extended periods, supporting its potential for chronic use.

Combination Potential:

  • Exploratory trials evaluated VE3 in combination with standard-of-care therapies like chemotherapy, yielding synergistic effects:
    • Enhanced tumor suppression compared to single-agent treatments.
    • Improved survival rates in animal models.
  • Data suggested that VE3’s mechanism of action complements existing therapies, reducing tumor resistance and increasing overall efficacy.

Achievements:

  1. Validated Efficacy:
    • Established VE3’s effectiveness in reducing tumor growth and metastasis in vivo.
  2. Safety Demonstrated:
    • Built a strong safety profile, critical for advancing to GLP toxicology studies.
  3. Optimized Dosage Strategies:
    • Refined dosing regimens for maximum efficacy with minimal toxicity.
  4. Combination Therapy Insights:
    • Highlighted VE3’s potential to serve as a synergistic agent with standard cancer treatments, broadening its clinical application.